JP2019518775A - カナバン病の治療 - Google Patents

カナバン病の治療 Download PDF

Info

Publication number
JP2019518775A
JP2019518775A JP2018567237A JP2018567237A JP2019518775A JP 2019518775 A JP2019518775 A JP 2019518775A JP 2018567237 A JP2018567237 A JP 2018567237A JP 2018567237 A JP2018567237 A JP 2018567237A JP 2019518775 A JP2019518775 A JP 2019518775A
Authority
JP
Japan
Prior art keywords
cells
ipscs
aspa
progenitor cells
npcs
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2018567237A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019518775A5 (https=
Inventor
シ，ヤンホン
チャオ，ジャンフェイ
リ，ウェンドン
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
City of Hope
Original Assignee
City of Hope
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by City of Hope filed Critical City of Hope
Publication of JP2019518775A publication Critical patent/JP2019518775A/ja
Publication of JP2019518775A5 publication Critical patent/JP2019518775A5/ja
Priority to JP2022199966A priority Critical patent/JP2023029376A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0623—Stem cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/602—Sox-2
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/603—Oct-3/4
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/604—Klf-4
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/606—Transcription factors c-Myc
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/608—Lin28
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/13—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from connective tissue cells, from mesenchymal cells
    • C12N2506/1307—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from connective tissue cells, from mesenchymal cells from adult fibroblasts
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Cell Biology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Transplantation (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Materials For Medical Uses (AREA)
JP2018567237A 2016-06-22 2017-06-22 カナバン病の治療 Pending JP2019518775A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022199966A JP2023029376A (ja) 2016-06-22 2022-12-15 カナバン病の治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662353515P 2016-06-22 2016-06-22
US62/353,515 2016-06-22
PCT/US2017/038853 WO2017223373A1 (en) 2016-06-22 2017-06-22 Treatment of canavan disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022199966A Division JP2023029376A (ja) 2016-06-22 2022-12-15 カナバン病の治療

Publications (2)

Publication Number Publication Date
JP2019518775A true JP2019518775A (ja) 2019-07-04
JP2019518775A5 JP2019518775A5 (https=) 2020-07-27

Family

ID=60784859

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018567237A Pending JP2019518775A (ja) 2016-06-22 2017-06-22 カナバン病の治療
JP2022199966A Pending JP2023029376A (ja) 2016-06-22 2022-12-15 カナバン病の治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022199966A Pending JP2023029376A (ja) 2016-06-22 2022-12-15 カナバン病の治療

Country Status (7)

Country Link
US (2) US20190307808A1 (https=)
EP (1) EP3474876A4 (https=)
JP (2) JP2019518775A (https=)
CN (1) CN110035764B (https=)
AU (1) AU2017281374A1 (https=)
CA (1) CA3028714A1 (https=)
WO (1) WO2017223373A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022076206A1 (en) * 2020-10-05 2022-04-14 City Of Hope Treatment of canavan disease
CN117999363A (zh) * 2021-08-17 2024-05-07 希望之城 用于无细胞的dna表观遗传的胃肠道癌检测以及治疗的组成物和方法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
JP2016507568A (ja) * 2013-02-06 2016-03-10 ユニバーシティー オブ ロチェスター ミエリン障害の治療のための誘導多能性細胞由来オリゴデンドロサイト前駆細胞

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2172963C (en) * 1993-09-29 2007-09-11 Reuben Matalon Aspartoacylase gene, protein, and methods of screening for mutations associated with canavan disease
WO2013120497A1 (en) * 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
PL2861733T3 (pl) * 2012-05-30 2021-04-19 Rowan University Terapia metaboliczna stresu oksydacyjnego w mózgu przez ukierunkowany neuronalny katabolizm kwasu n-acetyloasparaginowego

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016507568A (ja) * 2013-02-06 2016-03-10 ユニバーシティー オブ ロチェスター ミエリン障害の治療のための誘導多能性細胞由来オリゴデンドロサイト前駆細胞
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
HUMAN MOLECULAR GENETICS, 2008, VOL.17, NO.1, PP.R76-R83, JPN6022000288, ISSN: 0004679388 *
J GENE MED, vol. 13, JPN6021023098, 2011, pages 424 - 425, ISSN: 0004679387 *
NEUROCHEMICAL RESEARCH, 1999, VOL.24, NO.4, PP.507-513, JPN6022000286, ISSN: 0004849323 *
OFFICIAL JOURNAL OF THE AMERICAN SOCIETY OF GENE & CELL THERAPY, vol. 24, no. 3, JPN6022033582, February 2016 (2016-02-01), pages 430 - 446, ISSN: 0004849325 *
SCIENCE TRANSLATIONAL MEDICINE, 2012, VOL.4, NO.165, 165RA162, JPN6022000284, ISSN: 0004849322 *
SCIENCE TRANSLATIONAL MEDICINE, vol. 4, no. 165, JPN6022033584, 2012, pages 165 - 163, ISSN: 0004849324 *
THE JOURNAL OF GENE MEDICINE, 2011, VOL.13, PP.410-446, JPN6022000283, ISSN: 0004849321 *

Also Published As

Publication number Publication date
US20190307808A1 (en) 2019-10-10
WO2017223373A1 (en) 2017-12-28
JP2023029376A (ja) 2023-03-03
CA3028714A1 (en) 2017-12-28
EP3474876A4 (en) 2020-01-15
EP3474876A1 (en) 2019-05-01
AU2017281374A1 (en) 2019-01-03
CN110035764B (zh) 2023-08-15
US20240197789A1 (en) 2024-06-20
CN110035764A (zh) 2019-07-19

Similar Documents

Publication Publication Date Title
JP7277978B2 (ja) ミエリン障害の治療のための誘導多能性細胞由来オリゴデンドロサイト前駆細胞
Rubio et al. Genetically perpetuated human neural stem cells engraft and differentiate into the adult mammalian brain
Sandner et al. Neural stem cells for spinal cord repair
US10119123B2 (en) Cell fate conversion of differentiated somatic cells into glial cells
Kennea et al. Differentiation of human fetal mesenchymal stem cells into cells with an oligodendrocyte phenotype
US20240197789A1 (en) Treatment of canavan disease
US8962314B2 (en) Lateral ventricle cell compositions and use for treating neural degenerative diseases
JP6346629B2 (ja) 哺乳動物由来グリア制限前駆細胞を増大、同定、特徴付けおよび能力増強するための方法および組成物
Chen et al. Progress in dopaminergic cell replacement and regenerative strategies for Parkinson’s disease
Kim et al. Generation of human myogenic progenitors from pluripotent stem cells for in vivo regeneration
Beatriz et al. Revisiting cell and gene therapies in Huntington’s disease
US20240254440A1 (en) Methods to generate glial restricted neural progenitor cells and uses thereof
Surendran et al. Mouse neural progenitor cells differentiate into oligodendrocytes in the brain of a knockout mouse model of Canavan disease
US20230416689A1 (en) Treatment of canavan disease
Bonaventura et al. Induced pluripotent stem cells: an innovative patient-specific neurodegenerative disease modeling
Han et al. Stem Cell Therapy for ParkinsonTs
Griffin Ex vivo gene therapy for lysosomal storage disease using IPSC-derived neural stem cells
Ribeiro Cell Therapy in Brain Neurodegenerative Movement Disorders: A Clinical Perspective
Al-maqtari Approaches in the treatment of Parkinson's disease: a focus on stem cell-based therapies
Sandner et al. Stem Cell-Based Therapies for Spinal Cord Regeneration
WO2008124863A1 (en) Method of generating population of cells enriched for undifferentiated cells

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200610

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200610

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210615

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210915

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210922

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220111

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220408

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20220816